FDA is announcing a final rule that will improve the quality of safety reports and better protect people participating in clinical trials for drugs. The final rule clarifies the safety information that drug sponsors (drug manufacturers) must report to FDA for investigational new drug applications (INDs).

What is an IND?

An IND is the vehicle through which a sponsor advances to the stage of drug development known as clinical trials. Clinical trials test potential treatments in human volunteers to see whether these treatments should be approved for wider use in the general population.

Investigationaldrugs are new drugs that have not yet been approved by FDA or approved drugs that have not yet been approved for a new use, and are in the process of being tested for safety and effectiveness.

Why was this final rule established?

Sponsors submit drug safety information to FDA in an IND safety report. Because the drugs being tested are investigational and their adverse effects on people are not completely known, appropriate safety reporting is an important part of the clinical trial process. The final rule lays out clear definitions and standards to help ensure that critical safety information about an investigational new drug is accurately and rapidly reported.

How does FDA's final rule enhance the safety of participants in clinical trials?

The final rule will help sponsors decide whether an adverse event that occurs in a participant during a clinical trial is related to the investigational drug being tested, or if it is related to other health problems in the trial participants. Reports that focus on the most significant adverse events help FDA, sponsors, and clinical investigators better understand how to use the drug safely.

How does the final rule help sponsors determine when an adverse event needs to be reported rapidly to FDA?

The final rule defines new terms that help clarify when an adverse event should be reported to FDA rapidly - within 7 to 15 days. The rule explains that adverse events should be reported to FDA in an expedited manner only when there is a reasonable possibility that the drug caused the adverse event. The final rule also makes clear what information should not be reported to FDA because the information would not help the agency assess the safety of the drug being studied.

What are some of the other new requirements explained in this final rule?

The final rule enhances the protection of clinical trial participants by requiring rapid reports to FDA of certain safety information that had not previously been required. For example, findings from clinical or epidemiological studies that suggest a significant risk or serious, suspected adverse reactions that occur more frequently than anticipated must now be reported rapidly to FDA.

Another new requirement is that serious adverse events from bioavailability and bioequivalence studies, which are typically conducted for generic drugs, need to be reported rapidly to FDA.

Is this final rule consistent with requirements for clinical trials conducted outside of the United States?

Yes. Many clinical trials are conducted in countries outside the United States, so the revised definitions and reporting standards in this final rule are designed to be as consistent as possible with international definitions and standards. This should help lead to consistent, harmonized reporting for globally conducted clinical trials. Safety information from clinical studies conducted outside the United States can be evaluated along with information from U.S. clinical trials.

Will the new rule make the process of bringing new drugs to market slower and more burdensome?

No. In fact, the new rule should make the process more efficient. Reviewing and evaluating uninformative individual safety reports places a tremendous burden on the system without accompanying benefit. The new rule will reduce the current number of uninformative individual safety reports. This will improve the ability of sponsors, FDA, investigators, and institutional review boards (IRBs) to focus on the safety issues that affect patients and the public health. IRBs are groups of scientists, doctors, and lay people who review and approve clinical trials taking place within their jurisdiction before the trials can begin.